Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia

The recruitment status of this study is unknown because the information has not been verified recently.
Verified August 2010 by Shandong University.
Recruitment status was  Recruiting
Sponsor:
Collaborator:
National Natural Science Foundation of China
Information provided by:
Shandong University
ClinicalTrials.gov Identifier:
NCT01182662
First received: August 11, 2010
Last updated: August 30, 2010
Last verified: August 2010

August 11, 2010
August 30, 2010
August 2010
August 2013   (final data collection date for primary outcome measure)
  • SAA clinical symptoms [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Anemia symptoms, bleeding and infection will be mainly observed in every monthly after transplanting MSCs for one year.
  • The number of blood cells [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    The number of blood cells, which contains WBC, Neu, RBC, Hb,PLT and reticulocyte, will be mainly tested monthly after transplantion of MSCs for one year
  • Bone borrow hemocytology [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Bone borrow cytomorphologic examination will be tested in every 3 months after transplantion of MSCs for one year.
  • AA clinical symptoms [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Anemia symptoms, bleeding and infection will be mainly observed in every week after transplanting MSCs for one year.
  • The number of blood cells [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    The number of blood cells, which contains WBC, Neu, RBC, Hb and PLT, will be mainly tested in every month after transplanting MSCs for one year
  • Bone borrow hemocytology [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
    Bone borrow cytomorphologic examination will be tested in every 3 months after transplanting MSCs for one year.
Complete list of historical versions of study NCT01182662 on ClinicalTrials.gov Archive Site
Percentage of systemic T regulatory cell population and T lymphocyte subsets [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
Percentages of T regulatory cell population and T lymphocyte subsets in peripheral blood will be tested in every 3 months after transplanting MSCs for one year.
Percentage of systemic T regulatory population [ Time Frame: 1 year ] [ Designated as safety issue: Yes ]
Percentage of T regulatory cell population in peripheral blood will be tested in every 3 months after transplanting MSCs for one year.
Not Provided
Not Provided
 
Safety and Efficacy Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat Severe Aplastic Anemia
Phase II Study of Umbilical Cord/Placenta-Derived Mesenchymal Stem Cells to Treat SAA

The purpose of this study is to evaluate the safety and efficacy of mesenchymal stem cells (MSCs) derived from human umbilical cord/placenta at a dose of 1.0E+6 MSC/kg in subject for the therapy of severe aplastic anemia (SAA).

Severe aplastic anemia (SAA) is a condition that involves a low level of red blood cells, white blood cells, and platelets without evidence of another bone marrow disease. Patients with severe aplastic anemia produce too few blood cells, causing fatigue, easy bruising and bleeding, and susceptibility to infections. In many cases, the very low blood counts result from an autoimmune process. The patient's own immune system damages their stem cells in bone marrow.

Although immune-suppressing drugs, such as corticosteroids, CsA and ATG, have been used in the treatment of SAA, however, many studies have indicated that the overall response rate to these drugs is less than 60%. Addition, the severe side effects of these immune-suppressing drugs have also been observed. The management of SAA patients therefore remains unsatisfactory and targeted therapies are needed. Human MSCs isolated from human umbilical cord/placenta have been shown to have immunosuppressive, stimulating hematopoiesis and tissue repairing properties. This study will evaluate the safety and effectiveness of MSC transplantation in the SAA patients.

This study will last 2 to 3 years. Participants will be randomly assigned to receive either MSC transplant and CsA therapy (experimental group) or CsA therapy alone (control group). Patients will undergo MSC transplant at the start of the study on Day 0. After 3 months, patients will receive the second MSC transplantation. After six and twelve months from the first transplantation, patients will be evaluated.

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Single Blind (Outcomes Assessor)
Primary Purpose: Treatment
Aplastic Anemia
  • Other: Human umbilical cord-derived MSCs and cyclosporin A
    1.0E+6 MSC/kg, IV drop and repeat to apply in trimonthly for 2 cycle and cyclosporin A 5mg/kg po for 12 months
  • Other: cyclosporin A
    cyclosporin A 5mg/kg po for 12 months
  • Experimental: Human umbilical cord-derived MSCs and cyclosporin
    Human umbilical cord-derived MSCs at a dose of 1.0E+6 MSC/kg, repeated to apply in trimonthly for 2 cycle and CsA 5mg/kg po for 12 months
    Intervention: Other: Human umbilical cord-derived MSCs and cyclosporin A
  • Active Comparator: cyclosporine A
    cyclosporine A at a dose of 5 mg CsA/kg
    Intervention: Other: cyclosporin A
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
30
August 2013
August 2013   (final data collection date for primary outcome measure)

Inclusion Criteria:

  1. Patient age 18~80 years old with plan to infuse MSCs.
  2. Standard of diagnosis of aplastic anemia is according to Chinese domestic classification of AA for 1987.
  3. Patients must have an ECOG 0~2.
  4. No moderate or sever organ dysfunction: Ejection fraction>45%; Creatinine <176 umol/L.
  5. No active severe viral or fungus infection.
  6. Each patient must sign written informed consent.

Exclusion Criteria:

  1. Psychiatric condition that would limit informed consent.
  2. HIV positive
  3. Positive Pregnancy Test
  4. Patient has enrolled another clinical trial study within last 4 weeks.
Both
18 Years to 80 Years
No
Contact: chengyun zheng, Ph. D +86-531-85875635 chengyun.zheng@ki.se
China
 
NCT01182662
kongdx, No. 30670903
Yes
Chengyun Zheng, Department of Hematology of the 2nd Hospital of Shandong University
Shandong University
National Natural Science Foundation of China
Principal Investigator: chengyun zheng, Ph. D Department of Hematology of The 2nd Hospital of Shandong University
Shandong University
August 2010

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP